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Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Wednesday, May 24, 2023

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the Company’s investigational lentiviral-based gene therapy for Pyruvate Kinase Deficiency (PKD), a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown. RMAT designation was granted based on robust safety and efficacy data from the ongoing Phase 1 RP-L301 clinical trial and its potential to cure a life-threatening disease for which no curative therapies currently exist. The designation will provide the benefits of added FDA guidance and expedited review through the program’s development.

“Receiving RMAT designation from the FDA for RP-L301 is a major achievement in our pursuit to bring the first, potentially curative gene therapy treatment to patients living with PKD who have high unmet need. Notably, PKD has an estimated prevalence of up to 8,000 patients in the U.S. and Europe and represents one of the most significant patient opportunities in our LV hematology portfolio,” said Kinnari Patel, PharmD, MBA, President and Chief Operating Officer, Rocket Pharma. “Further, all four Rocket-sponsored programs with clinical data now have received RMAT designation from the FDA across both platforms, a unique showcase of our team’s ability both to select appropriate targets and develop gene therapies for them.”

Dr. Patel continued, “Results from the RP-L301 program demonstrate robust efficacy in both adult patients for up to 30 months with a highly favorable safety profile and were recently presented at ASGCT. The first pediatric patient has shown promising initial results similar to the adults, and enrollment has been completed in the Phase 1 study. We look forward to initiating the Phase 2 pivotal trial in the fourth quarter of 2023 as we continue to advance our world-class pipeline for patients facing such rare and devastating diseases.”

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition.

Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early FDA interactions to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review. In addition to RMAT, RP-L301 has also received Fast Track and Orphan Drug Designation.

Results from the Phase 1 program presented recently at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) demonstrate robust and sustained efficacy in both adult patients up to 30 months post-infusion demonstrated by normalized hemoglobin (from baseline levels in the 7.0-7.5 g/dL range), improved hemolysis parameters, red blood cell transfusion independence and improved quality of life with documented improvements via formal quality of life assessments. The safety profile appears highly favorable, with no RP-L301-related serious adverse events in either of the adult patients.

The first pediatric patient results suggest efficacy similar to the adult cohort with an initial greater than five-point increase in hemoglobin (from median baseline level of 7.9 g/dL). The infusion was well tolerated, with engraftment achieved at day +15, hospital discharge less than one month following infusion, and no RP-L301-related serious adverse events or red blood cell transfusion requirements following engraftment.

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